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帕金森病自体干细胞移植的进展

Advancements in Autologous Stem Cell Transplantation for Parkinson's Disease.

作者信息

Shi Jia-Xin, Zhang Ke-Zhong

机构信息

Department of Neurology, The First Affiliated Hospital of Nanjing Medical University, Nanjing, China.

出版信息

Curr Stem Cell Res Ther. 2024;19(10):1321-1327. doi: 10.2174/1574888X19666230907112413.

DOI:10.2174/1574888X19666230907112413
PMID:37691194
Abstract

Parkinson's disease (PD) is a progressive neurodegenerative disease marked by comparatively focal dopaminergic neuron degeneration in the substantia nigra of the midbrain and dopamine loss in the striatum, which causes motor and non-motor symptoms. Currently, pharmacological therapy and deep brain stimulation (DBS) are the primary treatment modalities for PD in clinical practice. While these approaches offer temporary symptom control, they do not address the underlying neurodegenerative process, and complications often arise. Stem cell replacement therapy is anticipated to prevent further progression of the disease due to its regenerative capacity, and considering the cost of immunosuppression and the potential immune dysfunctions, autologous stem cell transplantation holds promise as a significant method against allogeneic one to treat Parkinson's disease. In this review, the safety concerns surrounding tumorigenicity and complications associated with transplantation are discussed, along with methods utilized to evaluate the efficacy of such procedures. Subsequently, we summarize the preclinical and clinical studies involving autologous stem cell transplantation for PD, and finally talk about the benefits of autologous stem cell transplantation against allogeneic transplants.

摘要

帕金森病(PD)是一种进行性神经退行性疾病,其特征是中脑黑质中相对局限性的多巴胺能神经元变性以及纹状体中的多巴胺丧失,这会导致运动和非运动症状。目前,药物治疗和深部脑刺激(DBS)是临床实践中治疗帕金森病的主要方法。虽然这些方法能暂时控制症状,但它们并未解决潜在的神经退行性过程,且常常会出现并发症。由于干细胞替代疗法具有再生能力,有望预防疾病的进一步进展,并且考虑到免疫抑制的成本和潜在的免疫功能障碍,自体干细胞移植有望成为一种优于异体移植治疗帕金森病的重要方法。在这篇综述中,我们讨论了围绕移植相关的致瘤性和并发症的安全性问题,以及用于评估此类手术疗效的方法。随后,我们总结了涉及自体干细胞移植治疗帕金森病的临床前和临床研究,最后探讨了自体干细胞移植相对于异体移植的优势。

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本文引用的文献

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Parkinson's disease motor symptoms rescue by CRISPRa-reprogramming astrocytes into GABAergic neurons.通过 CRISPRa 重编程星形胶质细胞为 GABA 能神经元来拯救帕金森病运动症状。
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重复输注自体脂肪组织来源的干细胞治疗帕金森病。
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Preclinical Efficacy and Safety of a Human Embryonic Stem Cell-Derived Midbrain Dopamine Progenitor Product, MSK-DA01.人胚干细胞衍生中脑多巴胺前体细胞产品 MSK-DA01 的临床前疗效和安全性。
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Mesenchymal stem cells in Parkinson's disease: Motor and nonmotor symptoms in the early posttransplant period.帕金森病中的间充质干细胞:移植后早期的运动和非运动症状
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Grafts Derived from an α-Synuclein Triplication Patient Mediate Functional Recovery but Develop Disease-Associated Pathology in the 6-OHDA Model of Parkinson's Disease.源自α-突触核蛋白三倍体患者的移植物可介导帕金森病 6-OHDA 模型中的功能恢复,但会发展出与疾病相关的病理。
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